Biotech companies and industry groups are raising questions about the US FDA’s recent draft guidance on interpreting sameness of gene therapy products under its orphan drug regulations. The draft guidance, issued for comment in January, explains how the FDA intends to determine the sameness of two gene therapies intended for the same use or indication for the purposes of awarding orphan drug designation and exclusivity. While current regulations “do not elaborate on how the ‘same drug’ definition applies specifically to gene therapy products,” the FDA explains that it will consider the principal molecular structural features of the gene therapies to make its determination. Learn More